CN116096904A - 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途 - Google Patents

改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途 Download PDF

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CN116096904A
CN116096904A CN202080097613.5A CN202080097613A CN116096904A CN 116096904 A CN116096904 A CN 116096904A CN 202080097613 A CN202080097613 A CN 202080097613A CN 116096904 A CN116096904 A CN 116096904A
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acid sequence
seq
optionally
amino acid
aav
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肖恩·克拉克
卡伦·科扎尔斯基
图巴·居文-厄兹坎
安娜·特雷蒂亚科娃
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Swan Biotherapy Co ltd
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Swan Biotherapy Co ltd
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Animal Behavior & Ethology (AREA)
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  • Biophysics (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202080097613.5A 2019-12-31 2020-12-31 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途 Pending CN116096904A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962955667P 2019-12-31 2019-12-31
US62/955,667 2019-12-31
PCT/US2020/067664 WO2021138559A1 (en) 2019-12-31 2020-12-31 Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)

Publications (1)

Publication Number Publication Date
CN116096904A true CN116096904A (zh) 2023-05-09

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CN202080097613.5A Pending CN116096904A (zh) 2019-12-31 2020-12-31 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途

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US (2) US11779655B2 (https=)
EP (1) EP4085143B1 (https=)
JP (1) JP2023509443A (https=)
KR (1) KR20220128632A (https=)
CN (1) CN116096904A (https=)
AU (1) AU2020416291A1 (https=)
BR (1) BR112022013027A2 (https=)
CA (1) CA3166374A1 (https=)
IL (1) IL294278A (https=)
MX (1) MX2022008201A (https=)
WO (1) WO2021138559A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3228344A1 (en) * 2021-08-06 2023-02-09 Wuhan Neurophth Biotechnology Limited Company Composition and method for treating leber's hereditary optic neuropathy caused by nd4 mutation
KR20250009975A (ko) * 2022-05-12 2025-01-20 아브너진 인코포레이티드 재조합 파르보바이러스 생산을 위한 조성물 및 방법
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
AU2024315073A1 (en) * 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103717240A (zh) * 2011-06-10 2014-04-09 蓝鸟生物公司 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体
CN104470545A (zh) * 2012-05-04 2015-03-25 诺华股份有限公司 用于治疗视网膜营养不良的病毒载体
CN107287239A (zh) * 2016-04-11 2017-10-24 沈阳复明生物技术有限公司 一种用于视网膜色素变性的基因治疗载体及药物
WO2018136434A1 (en) * 2017-01-17 2018-07-26 Children's Medical Center Corporation Compositions and methods for diagnosing and treating peroxisomal diseases
CN108472264A (zh) * 2015-11-05 2018-08-31 通用医疗公司 编码用于治疗肾上腺脊髓神经病的abcd1的核酸序列的鞘内递送
CN109069669A (zh) * 2016-02-12 2018-12-21 康贝基因有限公司 载体
CN109843306A (zh) * 2016-08-19 2019-06-04 卡琳缪恩股份有限公司 使用自身互补型重组腺相关病毒治疗病症的方法和组合物

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8466271B2 (en) * 2008-07-23 2013-06-18 Boehringer Ingelheim Pharma Gmbh & Co, Kg Regulatory elements
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
EP2940131B1 (en) * 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
WO2015153889A2 (en) * 2014-04-02 2015-10-08 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
JP2018510160A (ja) * 2015-03-20 2018-04-12 ブルーバード バイオ， インコーポレイテッド ベクター製剤
CA3019315A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
PL3411484T3 (pl) * 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
EP3592137B1 (en) * 2017-03-10 2024-08-07 The Medical College of Wisconsin, Inc. Riboswitch modulated gene therapy for retinal diseases
WO2019060649A1 (en) * 2017-09-22 2019-03-28 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
MX2020004092A (es) * 2017-10-18 2021-01-08 Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas.
GB201817470D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103717240A (zh) * 2011-06-10 2014-04-09 蓝鸟生物公司 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体
CN104470545A (zh) * 2012-05-04 2015-03-25 诺华股份有限公司 用于治疗视网膜营养不良的病毒载体
CN108472264A (zh) * 2015-11-05 2018-08-31 通用医疗公司 编码用于治疗肾上腺脊髓神经病的abcd1的核酸序列的鞘内递送
CN109069669A (zh) * 2016-02-12 2018-12-21 康贝基因有限公司 载体
CN107287239A (zh) * 2016-04-11 2017-10-24 沈阳复明生物技术有限公司 一种用于视网膜色素变性的基因治疗载体及药物
CN109843306A (zh) * 2016-08-19 2019-06-04 卡琳缪恩股份有限公司 使用自身互补型重组腺相关病毒治疗病症的方法和组合物
WO2018136434A1 (en) * 2017-01-17 2018-07-26 Children's Medical Center Corporation Compositions and methods for diagnosing and treating peroxisomal diseases

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
HAERY L等: "Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation", FRONTIERS IN NEUROANATOMY, vol. 13, no. 1, 1 January 2019 (2019-01-01), pages 93, XP093057905, DOI: 10.3389/fnana.2019.00093 *
POWELL S K等: "Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy", DISCOVERY MEDICINE, 1 January 2015 (2015-01-01), pages 49 - 57 *
张德华: "蛋白质与酶工程", 30 September 2015, 合肥工业大学出版社, pages: 302 *

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US11779655B2 (en) 2023-10-10
IL294278A (en) 2022-08-01
MX2022008201A (es) 2022-10-18
BR112022013027A2 (pt) 2022-09-06
US20220175965A1 (en) 2022-06-09
CA3166374A1 (en) 2021-07-08
EP4085143A1 (en) 2022-11-09
EP4085143B1 (en) 2025-08-13
US20240009326A1 (en) 2024-01-11
JP2023509443A (ja) 2023-03-08
WO2021138559A1 (en) 2021-07-08
EP4085143A4 (en) 2024-02-28
US20220362403A2 (en) 2022-11-17
AU2020416291A1 (en) 2022-07-21
KR20220128632A (ko) 2022-09-21

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